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Retinal delivery of gene therapy via intravitreal injection of an engineered adeno-associated viral (AAV) vector

Intravitreal injection of an engineered AAV vector could enable retinal gene therapy. Directed evolution of AAV libraries identified an AAV variant that, after intravitreal injection in normal mice and monkeys, entered photoreceptors and other layers of the outer retina. In mouse models for X-linked retinoschisis or Leber's congenital amaurosis (LCA), intravitreal injection of the AAV variant expressing retinoschisis X-linked juvenile 1 (Rs1; Xlrs1) or retinal pigment epithelium-specific protein 65 kDa (Rpe65) decreased signs of disease and increased retinal function compared with injection of control AAV vectors expressing the respective genes. Future studies could include testing the AAV variant as a gene therapy vector in animal models for other congenital retinal diseases.

SciBX 6(28); doi:10.1038/scibx.2013.738
Published online July 25, 2013

Patented by The Regents of the University of California; licensed to an undisclosed entity; available for partnering

Dalkara, D. et al. Sci. Transl. Med.; published online June 12, 2013;
Contact: David V. Schaffer, University of California, Berkeley, Calif.
Contact: John G. Flannery, same affiliation as above